Dr. Andrew Garton, a resident in the Department of Neurological Surgery at Weill Cornell Medicine, is a co-first author of a significant new study that investigates patterns of disease progression in children with diffuse intrinsic pontine glioma (DIPG) — a deadly and treatment-resistant pediatric brain tumor.
Andrew Garton, MD PGY-7 Resident
The research, published in Neuro-Oncology Advances, highlights how a novel therapeutic approach may help improve local tumor control in a disease known for its poor prognosis.
The study, “Patterns of relapse in diffuse intrinsic pontine glioma after convection-enhanced delivery of 124I-omburtamab,” analyzed outcomes for 63 pediatric patients treated at Memorial Sloan Kettering Cancer Center.
Half the cohort received a novel form of targeted therapy known as convection-enhanced delivery (CED), in which the radiolabeled antibody omburtamab is infused directly into the tumor.
“Our goal was to determine whether CED could improve locoregional control of this devastating tumor,” said Dr. Garton. “While overall survival remained similar across groups, we found that patients who received CED were significantly less likely to experience tumor regrowth in the original tumor site and surrounding brainstem regions.”
Patients who received CED showed a lower cumulative incidence of local pontine progression and medullary relapse compared to those treated with radiation alone. These findings suggest that CED can help control disease within the critical areas of the brain targeted by the infusion, even if overall survival does not significantly improve with a single treatment.
The study also introduces a novel framework for analyzing radiographic patterns of recurrence in DIPG, information that could inform the design of future clinical trials and help refine targeted delivery approaches.
“This work is an essential step forward in understanding how locally delivered therapies can influence the natural history of DIPG,” said Dr. Mark M. Souweidane, vice chair of the Department of Neurological Surgery at Weill Cornell Medicine and senior author of the study. “Even in the absence of survival benefit, seeing improved control in the region of treatment gives us critical insight into how to improve delivery strategies, select patients, and guide the next generation of trials.”
Dr. Evan Bander, Director of Endoscopic Skull Base Surgery at Baptist Health Miami Neuroscience Institute and former neurosurgery resident at Weill Cornell Medicine, also contributed to this important research and was closely involved in early work surrounding convection-enhanced delivery during his residency.
DIPG remains one of the most difficult pediatric brain tumors to treat, with a median overall survival of less than one year. Traditional systemic therapies have shown limited efficacy due to the tumor's location in the brainstem and the inability of drugs to cross the blood-brain barrier.
“This work reinforces the importance of studying not only whether a treatment extends life, but how and where it may alter the disease process itself,” said Dr. Garton.
The full article is available here.